Correction of murine beta-thalassemia by gene transfer into the germ line

Science ◽  
1986 ◽  
Vol 233 (4769) ◽  
pp. 1192-1194 ◽  
Author(s):  
F Costantini ◽  
K Chada ◽  
J Magram
Keyword(s):  
Gene Transfer ◽  
Germ Line ◽  
Beta Thalassemia

Transgenic Mice: Gene Transfer into the Germ Line

Gene Transfer ◽  
1986 ◽  
pp. 189-221 ◽  
Author(s):  
Shirley M. Tilghman ◽  
Arnold J. Levine
Keyword(s):  
Gene Transfer ◽  
Transgenic Mice ◽  
Germ Line

No evidence of germ-line transmission by adenovirus-mediated gene transfer to mouse testes

2008 ◽  
Vol 89 (5) ◽  
pp. 1448-1454 ◽  
Author(s):  
Yoshiyuki Kojima ◽  
Yutaro Hayashi ◽  
Satoshi Kurokawa ◽  
Kentaro Mizuno ◽  
Shoichi Sasaki ◽  
...  
Keyword(s):  
Gene Transfer ◽  
Germ Line ◽  
Germ Line Transmission

Gene transfer into the Drosophila germ line

Science ◽  
1982 ◽  
Vol 218 (4570) ◽  
pp. 364-365 ◽  
Author(s):  
J. Marx
Keyword(s):  
Gene Transfer ◽  
Germ Line

Genetic Therapy for Beta-Thalassemia: From the Bench to the Bedside

Hematology ◽  
2010 ◽  
Vol 2010 (1) ◽  
pp. 445-450 ◽  
Author(s):  
Paritha Arumugam ◽  
Punam Malik
Keyword(s):  
Gene Therapy ◽  
Bone Marrow ◽  
Gene Transfer ◽  
Bone Marrow Cells ◽  
Genetic Disorder ◽  
Globin Gene ◽  
Retroviral Vectors ◽  
Beta Thalassemia ◽  
Great Success ◽  
Hematopoietic Stem

AbstractBeta-thalassemia is a genetic disorder with mutations in the β-globin gene that reduce or abolish β-globin protein production. Patients with β-thalassemia major (Cooley's anemia) become severely anemic by 6 to 18 months of age, and are transfusion dependent for life, while those with thalassemia intermedia, a less-severe form of thalassemia, are intermittently or rarely transfused. An allogeneically matched bone marrow transplant is curative, although it is restricted to those with matched donors. Gene therapy holds the promise of “fixing” one's own bone marrow cells by transferring the normal β-globin or γ-globin gene into hematopoietic stem cells (HSCs) to permanently produce normal red blood cells. Requirements for effective gene transfer for the treatment of β-thalassemia are regulated, erythroid-specific, consistent, and high-level β-globin or γ-globin expression. Gamma retroviral vectors have had great success with immune-deficiency disorders, but due to vector-associated limitations, they have limited utility in hemoglobinopathies. Lentivirus vectors, on the other hand, have now been shown in several studies to correct mouse and animal models of thalassemia. The immediate challenges of the field as it moves toward clinical trials are to optimize gene transfer and engraftment of a high proportion of genetically modified HSCs and to minimize the adverse consequences that can result from random integration of vectors into the genome by improving current vector design or developing novel vectors. This article discusses the current state of the art in gene therapy for β-thalassemia and some of the challenges it faces in human trials.

Gene transfer into the mouse germ-line

1982 ◽  
Vol 113 (S1) ◽  
pp. 219-226 ◽  
Author(s):  
Franklin Costantini ◽  
Elizabeth Lacy
Keyword(s):  
Gene Transfer ◽  
Germ Line
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